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Successful trial sees PROQS stock soar

Shares in Dutch pharmaceutical company ProQR Therapeutics soared after early-stage drug trials helped improve vision for sufferers of a rare form of childhood blindness.

The company’s shares more than doubled following the announcement of trial results during the first week of September, before continuing to surge the following week to hit a high of US$21.60 (AU$30.27). The stock was trading as low as US$3.30 (AU$4.62) at the start of the year.

Using its RNA-based drug QR-110, the company tested 10 people with a specific genetic mutation that caused Leber’s congenital amaurosis 10 – an eye disorder affecting at least 2,000 patients in Europe and the US, according to the company’s estimates.

After three months of treatment, about 60% of those tested showed a clinically meaningful response with improvements in vision and the ability to navigate, ProQR said in a statement.

There is currently no approved treatment for the condition, which affects the retina and causes severe visual impairment from infancy. However, the company is pushing toward mid-stage trials during the first half of 2019.

According to global investments research firm JMP Securities, the market is worth more than US$700 million (AU$980.9 m), as the only other potential competitor is the gene-editing treatment approach from Editas Medicine.

JMP’s analyst Ms Liisa Bayko told Reuters QR-110 now had a 60% probability of success, a huge jump from her previous estimate of 20%.

She also said the drug would likely be launched in the US in 2021, a year earlier than previously estimated, and that trial data had shown a “rapid and sustained benefit on every metric of vision assessed”.

“These results are very encouraging for our broader pipeline,” ProQR CEO Mr Daniel de Boer told Reuters, adding that the company was testing additional drugs with similar mechanisms.

The company is also developing treatments for other rare eye diseases, including Usher syndrome and Stargardt’s disease.